Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) Clinical Trial

Official title:

MPS VI Clinical Surveillance Program (CSP)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00214773
• Other study ID #: MPSVI CSP
• Status: Completed
• Start date: July 2005
• Est. completion date: May 1, 2020

Study information

• Verified date: January 2019
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 237
• Est. completion date: May 1, 2020
• Est. primary completion date: May 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria

All patients must meet the following criteria to qualify for enrollment in the CSP:

• Patient or patient's parent or legal guardian, if child is under 18 year old or is unable to consent, has provided a signed Patient Information and Authorization Form.

• Patient has laboratory results confirming a diagnosis of MPS VI disease based on detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood spots)and/or abnormality on the ARSB gene.

• Patient is willing to undergo general assessments to establish baseline data or permits physician to enter assessment data recorded prior to CSP entry if available in the patient's medical records. General assessments include: urinary GAG level, urinary protein level, serum sample for antibody levels, height, weight, and patient history.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis VI
• Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

Locations

Country	Name	City	State
Australia	Women and Children's Hospital	Adelaide	South Australia
Australia	Royal Children's Hospital, Brisbane	Brisbane	Queensland
Australia	Royal Children's Hospital	Parkville	Victoria
Australia	Princess Margaret Hospital for Children	Subiaco	Western Australia
Australia	Westmead Hospital	Wentworthville	New South Wales
Austria	LKH-Universitätsklinik Graz, Kinderklinik	Graz	Steiermark
Belgium	Institute of Pathology and Genetics, Metabolic Unit	Charleroi	Wallonie
France	Hôpital Femme Mère Enfant	Bron cedex	Rhône-alpes
France	Hopital Necker - Enfants Malades	Paris
Germany	Charité-Universitätsmedizin Berlin	Berlin
Germany	Universitätsklinikum Hamburg Eppendorf	Hamburg
Germany	Oberarzt Pädiatrie	Hannover	Niedersachsen
Germany	Universitatsklinikum Freiburg, Klinik II	Kammin	Mecklenburg-vorpommern
Germany	Universitätsmedizin	Mainz	Rheinland-Pfalz RP
Ireland	MidWestern Regional Hospital	Limerick
Italy	Azienda Ospedaliero Universitaria	Cibali	Catania
Italy	Università Milano Bicocca, Resp. Centro "Fondazione Mariani"	Monza
Italy	Department of Woman's and Child's Health	Padova	Veneto
Lithuania	Vilnius University Hospital, Santariskiu Klinikos	Vilnius
Netherlands	Rotterdam University Hospital - Sophia's Children's Hospital	Rotterdam
Portugal	Hospital de Sao Joao, Unidae de Doencas Metabolicas	Porto
Sweden	Astrid Lindgrens Children's Hospital	Stockholm
United Kingdom	Birmingham Children's Hospital, Steelhouse	Birmingham
United Kingdom	Queen Elizabeth Hospital, University Hospitals Birmingham NHS	Birmingham
United Kingdom	Great Ormond Street Hospital For Children, NHS Foundation Trust	London
United Kingdom	St Mary's Hospital, Willink Biochemical Genetics Unit	Manchester
United Kingdom	Salford Royal Hospital NHS Trust	Salford
United States	Fullerton Genetic Center	Asheville	North Carolina
United States	Johns Hopkins Univeristy School of Medicine	Baltimore	Maryland
United States	Baton Rouge Clinic	Baton Rouge	Louisiana
United States	Carolinas Medical Center	Charlotte	North Carolina
United States	Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Emory University	Decatur	Georgia
United States	Indiana University School of Medicine	Indianapolis	Indiana
United States	University of California, Irvine	Irvine	California
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Children's Health Care	Minneapolis	Minnesota
United States	University of Minnesota - Fairview University Medical Center	Minneapolis	Minnesota
United States	Tulane University Medical Center	New Orleans	Louisiana
United States	New York University	New York	New York
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	Children's Hospital and Research Center Oakland	Oakland	California
United States	St. Joseph's Healthcare	Paterson	New Jersey
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	University of Utah Medical Center	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Sioux Valey Children's Speciality Clinics	Sioux Falls	South Dakota

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Belgium,  France,  Germany,  Ireland,  Italy,  Lithuania,  Netherlands,  Portugal,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.		at least 15 years

External Links

•   BioMarin Pharmaceutical Inc Website